Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
OCU410 · 2 trials · 2 indications
Rate of change (slope) of square root-transformed GA lesion area (√mm²/year) from baseline to Month 12 in treated eyes compared to control eyes as assessed by fundus autofluorescence (FAF) measurements
The primary endpoint is safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.
Measured by letter score. A higher score represents better vision
Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).
| Arm | Type | Description |
|---|---|---|
| OCU410 | EXPERIMENTAL | OCU410 Single Sub Retinal Injection, 200 µL, 1×10\^10 vg/eye |
| Untreated Control Arm | NO_INTERVENTION | Control Arm (Untreated) |
| Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL): | EXPERIMENTAL | Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration. |
| Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL): | EXPERIMENTAL | Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration. |
| Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL): | EXPERIMENTAL | High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration. |
| Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm | EXPERIMENTAL | Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration. |
| Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm | EXPERIMENTAL | Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration. |
| Control Arm | NO_INTERVENTION | No Intervention Control Arm: Subject will not receive any active study intervention |
| Name | Type | Description |
|---|---|---|
| OCU410 | BIOLOGICAL | Subretinal Injection |
Inclusion Criteria: 1. Subjects 55 years of age or older at consent. 2. BCVA of ≥25 ETDRS letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen Equivalent) and ≤80 ETDRS Letters (20/25 Snellen Equivalent) in the study eye. 3. Fundus autofluorescence (FAF) im...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| AbbVie, Inc. | ABBV | 7 | PHASE3 | Surabgene Lomparvovec, RGX-314, ABBV-6628 |
| Ocular Therapeutix Inc | OCUL | 3 | PHASE3 | OTX-TKI |
| 4D Molecular Therapeutics, Inc. | FDMT | 3 | PHASE3 | 4D-150 |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE3 | Aflibercept, Pozelimab, Cemdisiran |
| Novartis AG Sponsored ADR | NVS | 5 | PHASE2 | Iptacopan, GT005, FWY003 |
| EyePoint, Inc. | EYPT | 2 | PHASE3 | EYP-1901 |
| Kodiak Sciences, Inc. | KOD | 1 | PHASE3 | Tarcocimab tedromer, Tabirafusp tedromer |
| Hoffmann-La Roche | RHHBY | 10 | PHASE3 | Ranibizumab, Susvimo, RO7965032, OpRegen, Faricimab |
| Outlook Therapeutics, Inc. | OTLK | 1 | PHASE3 | ONS-5010 |
| Adverum Biotechnologies, Inc. | ADVM | 4 | PHASE3 | Ixo-vec, ADVM-022 |
| Apellis Pharmaceuticals, Inc. | APLS | 1 | PHASE2 | APL-3007 |
| Amgen Inc. | AMGN | 1 | PHASE3 | ABP 938, Aflibercept |
| Johnson & Johnson | JNJ | 1 | PHASE2 | JNJ-81201887 |
| Ocugen Inc | OCGN | 1 | PHASE3 | OCU410 |
| Alcon AG | ALC | 1 | PHASE1 | AR-14034 |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR402663 |
| Arrowhead Pharmaceuticals, Inc. | ARWR | 1 | PHASE1 | ARO-033 |
| Bayer | BAYRY | 2 | - | Aflibercept |
OCU410 is being developed for the treatment of geographic atrophy secondary to dry age-related macular degeneration, an advanced form of dry AMD that causes progressive loss of retinal tissue and central vision. It is an investigational therapy and has not been approved for commercial use.
OCU410 targets RORA, the retinoic acid receptor-related orphan receptor alpha. RORA is involved in regulating pathways associated with retinal cell survival and inflammation. By acting on this target, OCU410 is designed to address the degenerative processes underlying geographic atrophy secondary to dry AMD.
OCU410 is being developed by Ocugen, Inc., a biotechnology company that trades on the Nasdaq under the ticker symbol OCGN. Ocugen is the sponsor of the clinical program evaluating OCU410 in patients with geographic atrophy secondary to dry age-related macular degeneration.
OCU410 is in Phase 3 clinical development. The Phase 3 study is currently recruiting participants, and the program also includes an earlier Phase 1 study that is active but no longer recruiting. OCU410 has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.
OCU410 is being evaluated in two registered clinical studies. The Phase 3 trial, NCT07770828, is recruiting 237 participants in the United States to assess efficacy, safety, and tolerability in geographic atrophy secondary to dry AMD. The Phase 1 study, NCT06018558, enrolled 60 participants and is active but not recruiting.
OCU410 is not approved; it remains an investigational therapy in clinical development. The FDA has granted it Regenerative Medicine Advanced Therapy (RMAT) designation, which is intended to expedite development and review of regenerative medicine therapies for serious conditions. Ocugen is currently conducting a Phase 3 trial to evaluate the treatment.